Cargo-selective and adaptive delivery of nucleic acid therapeutics by bola-amphiphilic dendrimers.

ABSTRACT: Nucleic acid therapeutics are becoming an important drug modality, offering the unique opportunity to address "undruggable" targets, respond rapidly to evolving pathogens, and treat diseases at the gene level for precision medicine. However, nucleic acid therapeutics have poor bioavailability and are chemolabile and enzymolabile, imposing the need for delivery vectors. Dendrimers, by virtue of their well-defined structure and cooperative multivalence, represent precision delivery systems. We synthesized and studied bola-amphiphilic dendrimers for cargo-selective and on-demand delivery of DNA and small interfering RNA (siRNA), both important nucleic acid therapeutics. Remarkably, superior performances were achieved for siRNA delivery with the second-generation dendrimer, yet for DNA deliver

SUBMITTER: Chen J 

PROVIDER: S-EPMC10214173 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature